Tasly's Innovative Drug PXT3003 Enters CDE Priority Review, Gaining International Recognition

VCBeat (WeChat: vcbeat) has learned that on March 29, Tasly (600535) announced that PXT3003, a new drug co-developed by Tasly and the French company Pharnext, was included in the “Public Notice of Drug Registration Applications Proposed for Inclusion in the Priority Review Program (27th Batch)” released by the Center for Drug Evaluation (CDE).

PXT3003 is the world’s first drug approved for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A), representing an undisputed first-in-class therapy. Its inclusion in the priority review pathway signifies a substantial reduction in both approval waiting time and overall registration timeline, paving the way for rapid advancement into clinical trials. Meanwhile, driven by its “four-in-one” R&D model, Tasly continues to launch new products and build an international innovative drug platform.

GF Securities believes that the development of PXT3003 is a typical example of collaborative R&D introducing overseas projects. In the future, Tasly will gradually realize an innovative product portfolio through global R&D filings and simultaneous launches, collaborate with leading international peers, and lay out strategies to track high-quality early-stage global projects for the production of high-quality products. Guotai Junan Securities analyzes that the market potential of PXT3003 is expected to reach RMB 500–1 billion.

PXT3003 was fast-tracked for priority review due to its status as an orphan drug and an innovative therapy. Developed by the French company Pharnext, this novel combination drug was identified through bio-network pharmacology analysis of Charcot-Marie-Tooth disease type 1A (CMT1A), screening over 2,000 already marketed drug molecules for their ability to downregulate PMP22 expression. It consists of a fixed-ratio combination of baclofen, naltrexone hydrochloride, and sorbitol, all administered at very low doses.

This drug is the world’s first therapy approved for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). CMT1A is a rare neurodegenerative disorder that causes significant patient suffering, and there are currently no effective treatments or medications available in clinical practice. PXT3003, which holds independent intellectual property rights, has been granted orphan drug designation by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and is an undisputed first-in-class innovative drug.

Currently, the drug is undergoing clinical trials globally. It has been granted a waiver for Phase I clinical studies in Europe and has completed Phase II clinical studies involving 80 patients. The efficacy and safety results from the Phase II trials indicate that PXT3003 possesses clear clinical value. The ongoing international multicenter Phase III clinical trial is progressing smoothly, with positive results obtained from two interim analyses—blinded variability analysis and futility analysis—completed in November 2017.

In May 2017, Tasly made a strategic investment of €20 million in Pharnext and jointly established Tasly Gene Network Company. Tasly holds a 65% equity stake in the company and possesses the commercialization rights for the PXT3003 project in Greater China (Mainland China, Hong Kong, Taiwan, and Macao). Additionally, Tasly holds exclusive license rights to all patents applied for and granted for PXT3003 in Greater China.

Announcement Discloses That Tasly Gene Network Co., Ltd. Has Invested Nearly RMB 23 Million in the Research and Development of PXT3003.

Tasly Gene Network Co., Ltd. applied on December 18, 2017, to conduct the international multi-center clinical trial of PXT3003 “in support of its registration in China,” and submitted its application for priority review and approval on February 6, 2018; the application is currently under the priority review process.

From the perspective of the drug approval process, the “Public Notice on Proposed Inclusion in the Priority Review List” issued by the Center for Drug Evaluation (CDE) of the China Food and Drug Administration will be posted for five days. If no objections are raised, PXT3003 will enter the priority review pathway, significantly shortening its approval waiting time and overall registration timeline, thereby enabling the product to advance rapidly into the clinical trial phase.

The inclusion of PXT3003 in the priority review program is a microcosm of Tasly’s recent achievements in building an international innovative drug platform. Leveraging its “four-in-one” R&D model—comprising independent R&D, product in-licensing, collaborative R&D, and investment with licensing options—the company has achieved comprehensive layout of its innovative R&D pipeline.

As of the end of 2017, Tasly had 74 products in its R&D pipeline, with several blockbuster new drugs gradually entering clinical trials and expected to be launched sequentially.

Through independent R&D, Tasly has 37 new drugs and generic drugs under development. Among them, the indication of Compound Danshen Dripping Pills for diabetic retinopathy is in Phase III clinical trials; the indications of Puyouke for ischemic stroke and acute pulmonary embolism are in Phase II clinical trials, and other key products are also applying for clinical trials.

Through product introductions, Tasly has 31 products in its R&D pipeline. These include leading candidates at various stages of development, such as PXT3003, Anmeimu Monoclonal Antibody (a core asset of Shanghai Saiyuan Technology), and a CD47 monoclonal antibody. The development of Anmeimu Monoclonal Antibody ranks among the top tier in China; it is currently undergoing Phase I clinical trials, with preliminary results demonstrating definitive efficacy and a safety profile superior to that of similar international marketed products.

Through collaborative R&D, the company has two products in its development pipeline. The first is the world’s first therapeutic hepatitis B vaccine using an adenovirus vector, co-developed with Transgene, a subsidiary of the French Mérieux Group. It is the only product of its kind under development in China, was included in the CDE’s Priority Review List in 2016, and is currently undergoing Phase I clinical trials. The second investigational product, T601, a recombinant oncolytic vaccinia virus injection, has submitted an Investigational New Drug (IND) application in China. This drug features dual functionalities of targeted oncolysis and targeted antibody therapy, significantly enhancing anti-tumor efficacy.

By investing in market priority rights, the company holds four products in its R&D pipeline. These include market priority rights for PegBio’s long-acting GLP-1 analog and GLP-1/glucagon dual receptor agonist, as well as sales priority rights for Jianya Bio’s insulin glargine and insulin lispro.

Among these, quality testing conducted by two authoritative third-party institutions in Europe and China demonstrated that Jianya’s third-generation insulin products exceeded the current European Pharmacopoeia standards in all comparative test items, showing consistency with the originator drugs from multinational pharmaceutical companies. The product purity and yield both surpass the current industry average for third-generation insulins domestically and internationally. In accordance with the current approval regulations, registration requirements, and quality standards of the European Union and the China National Medical Products Administration (NMPA), this insulin product will be submitted simultaneously for global dual filing as a biosimilar insulin to the European Medicines Agency (EMA) and the former China Food and Drug Administration (CFDA).

Tasly’s innovative drug R&D pipeline is flourishing across the board, a development closely tied to its sustained high level of investment. According to its 2017 annual report, R&D expenses saw a significant increase compared with 2016, reaching another record high.

According to the annual report, Tasly’s independent R&D investment in 2017 reached RMB 616 million, accounting for 9.04% of its pharmaceutical industry revenue; investments through other R&D approaches amounted to RMB 577 million, bringing total R&D expenditure to RMB 1.193 billion, which represented 17.5% of pharmaceutical industry revenue—a significant increase of 4.5 percentage points from the 13% R&D expense ratio recorded in 2016.

Market statistics show that traditional Chinese medicine (TCM) enterprises typically allocate 1%–2% of their revenue to R&D, with Tasly’s investment far surpassing that of its peers in the TCM sector. Across the entire pharmaceutical industry, the company ranks among the top five in terms of its R&D-to-sales ratio, establishing it as one of the most R&D-capable pharmaceutical enterprises in China.

Notably, Shanghai Tasly, a subsidiary of Tasly, is seeking a listing on the Main Board of the Hong Kong Stock Exchange. It holds the highest market capitalization among its peers planning to go public, further demonstrating Tasly’s commitment to building an international innovative industry platform.

Starting with traditional Chinese medicine (TCM) products, Tasly has gradually evolved into a pharmaceutical and biotechnology industrialization platform featuring the coordinated development of modern TCM, biologics, and chemical drugs, as well as the four-dimensional integration of R&D, manufacturing, marketing, and investment. Its international vision and global strategy have provided a valuable model for the development of China’s pharmaceutical and biotechnology enterprises.